CLINICAL TRIALS PROFILE FOR VIGABATRIN

505(b)(2) Clinical Trials for vigabatrin

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Hospices Civils de Lyon	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Institut National de la Santé Et de la Recherche Médicale, France	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	National Research Agency, France	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Orphelia Pharma	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.

All Clinical Trials for vigabatrin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00441896 ↗	A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms	Completed	Marinus Pharmaceuticals	Phase 2	2007-01-01	The study is a two period (8-10 days/period), incomplete cross-over in which successive cohorts of 9 subjects are randomized, in a 2:1 ratio, to 1 of 2 sequences, A and B. In each cohort, Sequence A, comprised of 6 subjects, receives ascending doses of ganaxolone during period 1 and ganaxolone (at the maximal dose attained in period 1) and ascending doses of placebo during period 2. Sequence B, comprised of 3 subjects, receives ascending doses of placebo during period 1 and receives the maximum dose of placebo and ascending doses of ganaxolone during period 2. The dosing level in each subsequent cohort will be based upon experience gained from previous cohorts.
NCT00506935 ↗	Assessment of GVG for the Treatment of Methamphetamine Dependence	Completed	University of California, Los Angeles	Phase 1	2006-07-01	The purpose of this study is to find out if GVG can reduce drug use and determine safety and effects of GVG when used together with methamphetamine. This study involves staying in the hospital for 21 days. Participants will receive either placebo or GVG, and a limited amount if methamphetamine will be injected on some study days. This study will enroll people that use methamphetamine. Participants will be compensated.
NCT00373581 ↗	Effects of Vigabatrin on Cocaine Self-Administration	Terminated	Novel Cocaine Pharmacotherapies	Phase 2	2006-04-01	The objective of this study is to determine if vigabatrin will decrease cocaine self-administration, cardiovascular effects, subjective effects and craving compared to placebo.
NCT00373581 ↗	Effects of Vigabatrin on Cocaine Self-Administration	Terminated	New York State Psychiatric Institute	Phase 2	2006-04-01	The objective of this study is to determine if vigabatrin will decrease cocaine self-administration, cardiovascular effects, subjective effects and craving compared to placebo.

Clinical Trial Conditions for vigabatrin

Condition Name

Condition Name for vigabatrin
Intervention	Trials
Infantile Spasms	5
Cocaine Dependence	5
Infantile Spasm	4
Cocaine Addiction	2

Condition MeSH

Condition MeSH for vigabatrin
Intervention	Trials
Spasms, Infantile	10
Spasm	10
Cocaine-Related Disorders	6
Muscle Cramp	4

Clinical Trial Locations for vigabatrin

Trials by Country

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−

Trials by Country for vigabatrin
Location	Trials
United States	95
France	2
Poland	2
Thailand	1
United Kingdom	1

Trials by US State

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−

Trials by US State for vigabatrin
Location	Trials
California	9
Texas	8
Florida	6
New York	6
Minnesota	5

Clinical Trial Progress for vigabatrin

Clinical Trial Phase

Clinical Trial Phase for vigabatrin
Clinical Trial Phase	Trials
Phase 4	5
Phase 3	4
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for vigabatrin
Clinical Trial Phase	Trials
Completed	11
Not yet recruiting	6
Terminated	5

Clinical Trial Sponsors for vigabatrin

Sponsor Name

Sponsor Name for vigabatrin
Sponsor	Trials
National Institute on Drug Abuse (NIDA)	4
Catalyst Pharmaceuticals, Inc.	4
Lundbeck LLC	3

Sponsor Type

Sponsor Type for vigabatrin
Sponsor	Trials
Other	45
Industry	15
NIH	5

Vigabatrin: Clinical Trials, Market Analysis, and Projections

Introduction

Vigabatrin, a GABA-aminotransferase (GABA-AT) inhibitor, is a crucial medication in the treatment of various seizure disorders, including infantile spasms and complex partial seizures. This article delves into the recent clinical trials, market analysis, and future projections for vigabatrin.

Clinical Trials Update

EPISTOP Trial

One of the significant clinical trials involving vigabatrin is the EPISTOP trial. This trial, conducted across 10 clinical sites in Europe and Australia, investigated the potential benefits of early treatment with vigabatrin to prevent seizures in infants with Tuberous Sclerosis Complex (TSC). The results, published in the Annals of Neurology, showed that early treatment with vigabatrin significantly delayed the onset of seizures in infants. At two years of age, seizures developed in only 52% of infants who received vigabatrin, compared to 84% of those who received conventional treatment. Additionally, no infantile spasms were observed in the vigabatrin group, and the risk of developing drug-resistant epilepsy was reduced[4].

Comparison with OV329

A recent pre-clinical study presented by Ovid Therapeutics highlighted the differences between vigabatrin and OV329, a next-generation anti-seizure medicine. The study found that OV329 did not accumulate in the retina, eyes, or brain tissues of mice, unlike vigabatrin, which preferentially accumulates in these tissues. This suggests that OV329 may offer a safer ocular profile compared to vigabatrin[1].

Market Analysis

Global Market Size and Growth

The vigabatrin market is anticipated to grow at a CAGR of 9.2% from 2023 to 2031. The market size is expected to expand from US$ XX million in 2023 to US$ XX million by 2031. This growth is driven by the increasing prevalence of epilepsy and seizure disorders, greater awareness and diagnosis of rare seizure disorders, and significant investments in neurology research and development[3].

Market Segmentation

The vigabatrin market is segmented by product (tablets and powder) and application (infantile spasms, partial-onset seizures, and refractory complex partial seizures). The market is also analyzed by region, with key markets including the United States, Europe, China, Japan, and Southeast Asia. Each region has its own consumption volume, value, and growth rate forecasts[3].

Competitive Landscape

The competitive landscape of the vigabatrin market includes several major players. These companies are profiled based on their product analysis, sales, price, revenue, and gross margin. The report also provides insights into the latest market dynamics, such as mergers, acquisitions, and investments, as well as market positioning and potential growth strategies[2].

Market Drivers and Opportunities

Increasing Prevalence of Epilepsy

The global rise in epilepsy and seizure disorders has significantly driven the demand for vigabatrin. This medication is particularly valuable for patients who are resistant to other treatments, making it a crucial component in the management of complex partial seizures and infantile spasms[3].

Greater Awareness and Diagnosis

Advances in medical knowledge and diagnostic capabilities have led to an increased understanding and diagnosis of rare and less treatable seizure disorders. This has expanded the market for vigabatrin as more cases are identified and treated with targeted therapies[3].

Investments in Neurology Research

Substantial investments in neurology research and development are buoying the vigabatrin market. These investments focus on developing new delivery mechanisms and improving the formulation of vigabatrin for better effectiveness and safety[3].

Personalized Medicine

The trend towards personalized medicine is also influencing the vigabatrin market. Healthcare is increasingly adopting personalized approaches based on genetic, metabolic, and lifestyle factors, which can lead to more precise and effective treatment outcomes for patients with epilepsy[3].

Advancements in Drug Delivery Systems

Continuous developments in drug delivery systems are enhancing the bioavailability and absorption of vigabatrin, reducing adverse effects, and maximizing therapeutic effectiveness. Innovations such as controlled release tablets and new dosing methods are broadening the use of the drug and improving patient compliance[3].

Market Challenges and Limitations

Ocular Accumulation

One of the significant challenges associated with vigabatrin is its tendency to accumulate in ocular tissues, which can lead to visual field defects. This issue is highlighted by the contrast with OV329, which does not accumulate in these tissues[1].

Side Effects

While the EPISTOP trial showed that early treatment with vigabatrin delayed the onset of seizures without major side effects, other studies have noted potential risks such as visual field defects and the possibility of neurodevelopmental delays[4].

Market Projections

Regional Growth

The vigabatrin market is expected to grow significantly in various regions, including the United States, Europe, China, Japan, and Southeast Asia. Emerging markets, particularly in developing regions, are anticipated to open up new growth opportunities as healthcare systems improve and focus on total care for neurology[3].

Forecast by Type and Application

The global vigabatrin market is forecasted to grow in terms of consumption volume, value, and price by type (tablets and powder) and application (infantile spasms, partial-onset seizures, and refractory complex partial seizures). The market is expected to expand as new delivery mechanisms and formulations are developed[3].

Innovative Research and Adjunctive Therapy

Combination Therapy

Research is being directed towards the complementary use of vigabatrin with other antiepileptic agents to optimize effectiveness and create new therapeutic avenues. This could broaden the indications for vigabatrin and enhance its role in multi-drug regimens[3].

Key Takeaways

• Clinical Trials: The EPISTOP trial demonstrated the efficacy of early vigabatrin treatment in delaying the onset of seizures in infants with TSC.
• Market Growth: The vigabatrin market is expected to grow at a CAGR of 9.2% from 2023 to 2031, driven by increasing prevalence of epilepsy and investments in neurology research.
• Market Segmentation: The market is segmented by product and application, with key regions showing significant growth potential.
• Challenges: Ocular accumulation and potential side effects are key challenges associated with vigabatrin.
• Projections: The market is forecasted to grow in various regions, with emerging markets offering new opportunities.

FAQs

What is the current status of the EPISTOP trial?

The EPISTOP trial found that early treatment with vigabatrin significantly delayed the onset of seizures in infants with Tuberous Sclerosis Complex (TSC) without major side effects[4].

How does vigabatrin compare to OV329 in terms of ocular safety?

Vigabatrin preferentially accumulates in ocular tissues, whereas OV329 does not accumulate in these tissues, suggesting a safer ocular profile for OV329[1].

What are the key drivers of the vigabatrin market?

The key drivers include the increasing prevalence of epilepsy, greater awareness and diagnosis of rare seizure disorders, and significant investments in neurology research and development[3].

What are the potential side effects of vigabatrin?

Potential side effects include visual field defects and the possibility of neurodevelopmental delays[4].

How is the vigabatrin market expected to grow in the future?

The market is expected to grow at a CAGR of 9.2% from 2023 to 2031, with significant growth potential in emerging markets[3].

Sources

1. Ovid Therapeutics Presents Pre-Clinical Study Results. GlobeNewswire, 2024.
2. Global Vigabatrin Consumption Market Research Report 2013-2025. Maiaresearch, 2024.
3. Vigabatrin Market - Size and Forecast [2024-2031]. The Insight Partners, 2024.
4. Positive results in vigabatrin trial to prevent infant seizures. Tuberous Sclerosis Alliance, 2021.